PHILADELPHIA—Two research teams from the Perelman School of Medicine at the University of Pennsylvania are among the recipients of the 2017 Clinical Research Achievement Award. Charles P. O’Brien, MD, PhD, the founding director of the Center for Addiction Treatment at Penn Medicine and holder of the Kenneth Appel Chair in Psychiatry is being recognized for creating the first U.S. large-scale clinical trial of extended-release naltrexone for treating opioid addiction. Aimee S. Payne, MD, PhD, the Albert M. Kligman Associate Professor of Dermatology, and Michael C. Milone, MD, PhD, Assistant Professor of Pathology and Laboratory Medicine, are being recognized for their research using chimeric autoantibody receptor (CAAR) T cells as a targeted therapy of autoimmunity.
The Clinical Research Forum recognized the year’s 10 most outstanding research papers written by teams from across the nation during its annual meeting and awards ceremony in Washington, D.C., on April 18.
O’Brien is being acknowledged for his research around a novel approach to opioid addiction treatment. Previously, the standard treatment for opioid addiction was methadone, an agonist drug that patients used daily to reduce the "high" caused from using opiates and to reduce cravings and withdrawal symptoms. Instead of daily doses of methadone, O’Brien and his colleagues used a completely different pharmacological strategy giving monthly injections of naltrexone, an opioid receptor antagonist drug that prevents the body from responding to opioids. Patients participating in the trial had a history of opioid addiction and were recently released from criminal justice restrictions, making them the perfect candidates for the antagonist therapy. The protocol was conducted at five sites in the Northeast with Penn as the coordinating site. After 24 weeks of treatment, patients randomly assigned to naltrexone experienced a longer median time to relapse and a lower rate of relapse than those assigned to usual treatment. Also, in the naltrexone group, there were no opioid overdoses. In contrast, there were seven overdoses in the comparison group. O’Brien reported his findings in a paper that was published in the New England Journal of Medicine.
Payne and Milone, along with postdoctoral fellow Christoph T. Ellebrecht, MD, were honored for their work developing an innovative approach to re-engineer chimeric antigen receptor (CAR) T cells to treat autoimmunity. Payne’s team devised a new method to remove the subset of antibody-making cells that cause an autoimmune disease, without harming the rest of the immune system. The autoimmune disease the team studied is called pemphigus vulgaris (PV), a condition in which a patient’s own immune cells attack a protein called desmoglein-3 (Dsg3) that normally adheres skin cells. Current therapies for autoimmune disease, such as prednisone and rituximab, suppress large parts of the immune system, leaving patients vulnerable to potentially fatal opportunistic infections and cancers. Payne’s team of researchers demonstrated the new technique by successfully treating an otherwise fatal autoimmune disease in a mouse model, without apparent off-target effects, which could harm healthy tissue. The results are published online in Science. The results of Payne and Milone’s study offer hope to patients living with pemphigus, a rare orphan disease that has no FDA-approved therapies.
Formed in 1996, the Clinical Research Forum convenes annually to allow industry leaders to discuss issues facing the field, best practices, and promote understanding and support for clinical research and its impact on health and healthcare. More information is available at www.clinicalresearchforum.org.
Penn Medicine is one of the world’s leading academic medical centers, dedicated to the related missions of medical education, biomedical research, excellence in patient care, and community service. The organization consists of the University of Pennsylvania Health System and Penn’s Raymond and Ruth Perelman School of Medicine, founded in 1765 as the nation’s first medical school.
The Perelman School of Medicine is consistently among the nation's top recipients of funding from the National Institutes of Health, with $550 million awarded in the 2022 fiscal year. Home to a proud history of “firsts” in medicine, Penn Medicine teams have pioneered discoveries and innovations that have shaped modern medicine, including recent breakthroughs such as CAR T cell therapy for cancer and the mRNA technology used in COVID-19 vaccines.
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